Serious flaws in rare disorders medicine fund proposal
Patient advocates are rallying support for submissions to Pharmac about the U-turn they have made on medicine funding
for ultra-rare disorders. After repeated refusal to fund novel and expensive treatments for disorders that affect mostly
just a handful of cases in New Zealand, Pharmac have announced a special $5 Million fund so they do get a chance of
being funded here.
“That’s one great step forward”, says executive director of the NZ Organisation for Rare Disorders, John Forman, “but it
is a long way from a serious solution to lack of access to these vital medicines”.
NZORD says the initial proposal from Pharmac suggested that the woefully inadequate budget was the major problem that
would make it extremely difficult to get fair access across 7 diseases and estimated 85 patients needing treatment.
“There needs to be $20 to $25 million per year invested in this area, rather than just $5 million, but we were still
keen to work with Pharmac to try and ensure the most equitable distribution of the limited funds across these patient
groups,” says Forman.
“However the formal consultation document shows serious flaws in the detail of the fund, which makes it even more
unlikely that any of the patients will get a fair shot at treatment. These patients face triple-jeopardy in the way
Pharmac has structured the criteria for the fund.”
“The first problem is the restrictive rules proposed by Pharmac, setting a higher efficacy threshold for these
treatments for ultra-rare diseases, than is expected for medicines for common diseases. Secondly, Pharmac’s special
committee for rare disorders has already signalled it will resist any acceptance of weaker evidence for them, even
though this is invariably the case for such rare conditions. The third problem is how the paper makes it clear there
will still be comparison of costs with medicines for common diseases, despite the obvious higher unit cost of
development, and widespread acceptance in other jurisdictions that such treatments will not meet standard
cost-effectiveness comparisons.” says Forman.
NZORD now suspects strongly that this system has been set up to fail, as reflected in the earlier assessment that the
proposal was more likely a device to dampen debate over the election period, than a serious attempt to bring in an
orphan drugs access programme.
NZORD is working with support groups for Muscular Dystrophy, Cystic Fibrosis, Lysosomal diseases, and Paroxysmal
Nocturnal Haemoglobinuria, in an attempt to obtain subsidy of 8 new medicines for about 85 NZ patients who are currently
left untreated.
Submissions on Pharmac’s consultation document close on Friday 25th July.